Insights+: The US FDA New Drug Approvals in June 2020

The US FDA has approved multiple NDAs and BLAs in Jun 2020, leading to treatments for patients and advances in the health care industry. The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 51 novel products so far in 2020, including 8 in Jun 2020. Additionally, last year in 2019, the US FDA has approved 48 novel products. PharmaShots has compiled a list of 8 new drugs approved by the US FDA in Jun 2020.

1. Viela's Uplizna (inebilizumab-cdon) Received the US FDA's Approval to Treat Neuromyelitis Optica Spectrum Disorder (NMOSD)

Published: Jun 11, 2020 | Tags: Viela, Uplizna, inebilizumab-cdon, Received, the US FDA, Approval, Treat, Neuromyelitis Optica Spectrum Disorder, NMOSD

The approval was based on N-MOmentum trial which involved assessing of Uplizna (300mg) vs placebo in 213 patients in the ratio (3:1) at day 1 to day 15 who are anti-AQP4 antibody-positive/negative
The study resulted in anti-AQP4 antibody-positive group remained relapse-free (89% vs 58%) including reductions in NMOSD-related hospitalizations with safety and tolerability profile
Uplizna (inebilizumab-cdon) is a humanized CD19-directed mAb and is a novel B cell depleter approved for the treatment of NMOSD in adults who are anti-aquaporin-4 (AQP4) antibody positive

2. ViiV Healthcare's Tivicay PD (dolutegravir) Received the US FDA's Approval for Children Living with HIV

Published: Jun 12, 2020 | Tags: ViiV Healthcare, Tivicay PD, dolutegravir, Receives, the US FDA, Approval, Children, Living, HIV

The approval is based on ongoing P1093 (NCT03016533) and ODYSSEY (PENTA20, NCT02259127) studies in pediatric patients aged 4 weeks to 18 years, while the second study involves 2L pediatric patients aged four weeks to 18 years
The P1093 study was conducted by IMPAACT network in the USA, Brazil, Thailand, South Africa, Zimbabwe, Kenya and Tanzania and ODYSSEY by the PENTA network in Europe, South America, Thailand, Uganda, Zimbabwe, and South Africa
Tivicay and Tivicay PD are a human immunodeficiency virus type 1 (HIV-1) integrase strand transfer inhibitor (INSTI) used in combination with antiretroviral agents to treat HIV-1 infection in adults and in pediatric patients aged at least 4 weeks and weighing at least 3 kg

3. Pharma Mar's Zepzelca (lurbinectedin) Received the US FDA's Accelerated Approval for Adult Patients with Metastatic Small Cell Lung Cancer (SCLC)

Published: Jun 15, 2020 | Tags: Pharma Mar, Zepzelca, lurbinectedin, Received, the US FDA, Accelerated Approval, Adult, Patients, Metastatic Small Cell Lung Cancer, SCLC

The approval was based on P-II trial assessing Zepzelca in 105 adults Ultragenyx with SCLC at 26 hospitals in the US and EU
The study resulted in ORR was 35%, median response duration 5.3 mos., as per investigator assessment using RECIST 1.1 and as per independent review committee, ORR was 30%, median response duration 5.1 months. Project Orbis conducted the review which is an initiative of the FDA Oncology Center of Excellence
Zepzelca is an alkylating agent that binds to DNA and interferes with transcription factors and controls cancer cell growth. The product has also received the US FDA's ODD for SCLC and priority review to this application

4. Ultragenyx's Dojolvi (UX007/triheptanoin) Received the US FDA's Approval to Treat Long-Chain Fatty Acid Oxidation Disorders

Published: Jun 15, 2020 | Tags: Ultragenyx, Dojolvi, UX007/triheptanoin, Receives, US FDA, Approval, Treat, Long-Chain, Fatty Acid, Oxidation Disorders

The FDA approved Dojolvi as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD)
The company expects its availability in the next 30 days while for its access the Ultragenyx's UltraCare program will help patients and caregivers to understand insurance coverage and assist them in finding financial support for Ultragenyx medicines
Ultragenyx is a highly purified, pharmaceutical-grade, odd-carbon medium-chain triglyceride consisting of three 7-carbon fatty acids on a glycerol backbone created via a multi-step chemical process

5. Eli Lilly's Lyumjev (insulin lispro-aabc injection) Received the US FDA's Approval for Type 1 and Type 2 Diabetes

Published: Jun 15, 2020 | Tags: Eli Lilly, Lyumjev, insulin lispro-aabc injection, Receives, US, FDA, Approval, Type 1, Type 2, Diabetes

The approval is based on P-III PRONTO-T1D and PRONTO-T2D studies assessing Lyumjev (insulin lispro-aabc injection, 100 units/mL) and Humalog (insulin lispro injection, 100 units/mL), both in combination with either insulin glargine or insulin degludec in adults with T1D and T2D, respectively
Both the studies resulted in meeting its 1EPs of non-inferior A1C reduction @26wks., when both the therapies dosed at mealtime, Lyumjev demonstrated a superior reduction in blood glucose spikes at both 1hr. & 2hrs. after a test meal, safety & tolerability profile is similar for both
Lyumjev is a novel formulation of insulin lispro, developed to speed the absorption of insulin into the bloodstream and reduce A1C levels and is contraindicated during episodes of hypoglycemia and in patients with hypersensitivity to insulin lispro or one of the excipients in Lyumjev

6. Evoke's Gimoti (metoclopramide) Received the US FDA's Approval to Relief Symptoms in Adults with Acute and Recurrent Diabetic Gastroparesis

Published: Jun 19, 2020 | Tags: Evoke, Gimoti, metoclopramide, Receives, the US FDA, Approval, Relief Symptoms, Adults, Acute, Recurrent Diabetic Gastroparesis

The US FDA's approval allows Evoke to access its existing $5M line of credit from Eversana for manufacturing and commercialization of Gimoti
The approval will serve as a novel treatment for gastroparesis with expected commercialization in Q4'20
Gimoti nasal spray is the novel nasally administered product while Eversana will be responsible for its launch

7. Zogenix's Fintepla (fenfluramine) Received the US FDA's Approval for the Treatment of Seizures Associated with Dravet Syndrome

Published: Jun 26, 2020 | Tags: Zogenix, Fintepla, fenfluramine, Received, US, FDA, Approval, Treatment, Seizures, Associated, Dravet Syndrome

The US FDA's approval was based on two P-III studies assessing Fintepla vs PBO in patients aged =2yrs. with Dravet syndrome and a safety data from an OLE trial in which many patients received Fintepla for up to 3yrs.
In addition to the existing treatment regimens, the therapy demonstrated a reduction in the frequency of monthly convulsive seizure in patients whose seizures were not controlled on one or more antiepileptic drugs
The company will launch the therapy through restricted distribution program, called the Fintepla REMS Program, and is expected to be available through Zogenix's specialty pharmacy partner by July'20

8. Roche's Phesgo (fixed-dose combination of Perjeta and Herceptin) Received the US FDA's Approval for Early and Metastatic HER2-Positive Breast Cancer

Published: Jun 30, 2020 | Tags: Roche, Phesgo, fixed-dose combination, Perjeta, Herceptin, Received, US, FDA, Approval, Early, Metastatic, Her2-Positive Breast Cancer

The approval was based on P-III FeDeriCa study which involved assessing of Phesgo (SC) + CT vs Perjeta (pertuzumab, IV) & Herceptin (trastuzumab, IV) + CT in 500 patients with HER2+ve early breast cancer treated in the neoadjuvant (before surgery) and adjuvant (after surgery) settings evaluated PK, efficacy, and safety
The study met its 1EPs showing non-inferior levels of Phesgo (SC) vs Perjeta (IV) in the blood plus no cardiac toxicity was demonstrated also the safety profile was comparable including no new safety signals. Additionally, in the P-II PHranceSCa study, 85% (136/160) patients preferred SC vs IV administration involving less time in the clinic with more comfort
Phesgo is an (FD, SC) formulation which involves a combination of pertuzumab + trastuzumab developed using Halozyme Therapeutics Enhanze drug delivery technology and administered within 8 mins for initial dosing and 5 mins for maintenance dosing